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Overview: Aqvesme is a medicine used to treat anemia in certain people with alpha- or beta-thalassemia. Both headache and trouble sleeping are common side effects. Aqvesme is only available through a restricted program because of the risk of liver problems. Keep all appointments to have your blood checked. Tell your health care provider right away if you get nausea, stomach pain, appetite loss, dark urine, or yellowing of the whites of your eyes or skin.

Uses

What is Aqvesme used for?

Aqvesme (mitapivat) is commonly used to treat anemia in certain people with alpha- or beta-thalassemia, which is an inherited blood disorder.

Navigating Anemia: What to Know About Aqvesme for Thalassemia

Aqvesme may also be used for other conditions as determined by your health care provider.

How does Aqvesme work (mechanism of action)?

If you have thalassemia, you do not make enough of a protein called hemoglobin. Hemoglobin is made of 2 parts: alpha and beta. If one of these parts is not being made, you do not have enough parts to make normal amounts of hemoglobin. You have alpha-thalassemia if you have low amounts of alpha hemoglobin. You have beta-thalassemia if you have low amounts of beta hemoglobin.

Hemoglobin is found in your red blood cells and carries oxygen throughout your body. When you do not have enough alpha or beta hemoglobin, your red blood cells are not made well and do not last as long as they normally would. When you do not have enough red blood cells, you have anemia. You may feel tired if you do not get enough oxygen delivered to your tissues. You may need regular blood transfusions.

Aqvesme works by increasing the activity of a protein called pyruvate kinase in red blood cells. Increased activity is thought to help red blood cells in many ways, so that they can develop better and last longer. 

Aqvesme may increase hemoglobin and reduce tiredness and the need for blood transfusions. 

How is Aqvesme supplied (dosage forms)?

Aqvesme is available as a 100 mg oral tablet that is swallowed.

How should I store Aqvesme?

Aqvesme blister wallets should be stored in the original carton in a refrigerator, between 36 F and 46 F (2 C and 8 C). It can be stored at room temperature, between 68 F and 77 F (20 C and 25 C) for up to 3 months. Store in a cool, dry place.

If you remove Aqvesme from the refrigerator, write the discard (throw away) date (3 months later) on either the blister wallet or carton. Throw away any medicine by the expiration date or the discard date.

Side Effects

What are the most common side effects of Aqvesme?

The most common side effects of Aqvesme are listed below. Tell your health care provider if you have any of these side effects that bother you.

  • Headache
  • Trouble sleeping

There may be other side effects of Aqvesme that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Aqvesme?

While less common, the most serious side effects of Aqvesme are described below, along with what to do if they happen.

Severe Allergic Reactions. Aqvesme may cause allergic reactions, which can be serious. Stop taking Aqvesme and get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching or skin rash
  • Bumps on the skin called hives that can be red, pink, white, or brown depending on your skin tone
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Liver Damage. Liver damage, also called hepatotoxicity, can happen when taking Aqvesme. Your health care provider will likely do blood tests to check for liver problems before and during treatment with this medicine, especially during the first 6 months of use or if you get symptoms of liver damage. Keep all appointments to have your blood checked. Call your health care provider right away if you have any of the following symptoms of liver damage.

  • Nausea or vomiting
  • Stomach or belly pain
  • Fever
  • Weakness or unusual tiredness
  • Itching
  • Loss of appetite
  • Light-colored poop
  • Dark-colored urine (pee)
  • Your skin or the whites of your eyes turning yellowish in color (also called jaundice)

Warnings & Precautions

Who should not use Aqvesme?

Allergies to Ingredients. People who are allergic to any of the following should not take Aqvesme.

  • Aqvesme
  • Mitapivat
  • Pyrukynd
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Aqvesme.

What should I know about Aqvesme before using it?

Do not take Aqvesme unless it has been prescribed to you by a health care provider. Take it as prescribed.

Do not share Aqvesme with other people, even if they have the same condition as you. It may harm them.

Keep Aqvesme out of the reach of children. Since this medicine should be kept in a refrigerator, take special precautions to keep it away from children who also use the refrigerator.

Swallow Aqvesme whole. Do not split, crush, chew, or dissolve the tablets.

Aqvesme is only available through a special program, from certain pharmacies, and to certain people. It is important that you keep all of your appointments, including labs or other testing, while taking Aqvesme. If you have any questions about this program you can go to www.aqvesmerems.com or call 800-625-9951.

Your health care provider may pause or permanently stop Aqvesme if you get certain side effects. Tell your health care provider right away about any new or worsening side effects.

If you use birth control that contains hormones like ethinyl estradiol, talk with your health care provider. You may need to use a different type of birth control or also use a nonhormonal method like condoms. 

Hormonal birth control with the exception of an intrauterine device (IUD) containing levonorgestrel may not work well when you take Aqvesme and for 28 days after your last dose. 

What should I tell my health care provider before using Aqvesme?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them know if Aqvesme is right for you.

In particular, make sure that you discuss any of the following.

Liver Problems. Tell your health care provider if you have any liver problems. Make sure that you let your health care provider know if you have cirrhosis, even if you have no symptoms. Aqvesme can cause liver injury and is not recommended for use by patients with any degree of cirrhosis (Child-Pugh Class A, B, or C).

Pregnancy. It is not known if or how Aqvesme could affect pregnancy or harm a fetus.  If you can become pregnant, talk with your health care provider about what birth control to use. Tell your health care provider right away if you are pregnant, think you may be pregnant, or plan to become pregnant.

Breastfeeding. It is not known if Aqvesme passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed. Your health care provider will let you know if you should take Aqvesme while breastfeeding.

How can I get more information about Aqvesme?

For more information about Aqvesme, you can visit the drugmaker’s website at www.aqvesme.com/thalassemia or call them at 877-772-4467.

Interactions

Does Aqvesme interact with foods or drinks?

There are no known interactions between Aqvesme and foods or drinks.

It is unknown if drinking alcohol will affect Aqvesme. Both alcohol and Aqvesme can cause trouble sleeping. It is best to limit the amount of alcohol that you drink.

Does Aqvesme interact with other medicines (drug interactions)?

Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

In particular, make sure that you discuss if you are using any of the following before taking Aqvesme.

  • A birth control pill, patch, vaginal ring, implant, or injection that contains hormones
  • Rifampin (Rifadin), which is a medicine commonly used to treat an infection called tuberculosis
  • Fluconazole (Diflucan), itraconazole (Sporanox, Tolsura), or ketoconazole, which is a medicine used for fungal infections

Many other medicines may alter the blood levels of Aqvesme, and vice versa. Tell your health care provider about all medicines that you take or have recently taken.

This may not be a complete list of medicines that can interact with Aqvesme. Always check with your health care provider.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Aqvesme?

If you or someone else has used too much Aqvesme, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Aqvesme?

If you miss a dose and it has been 4 hours or less, take it as soon as you remember. If you miss a dose by more than 4 hours, skip the missed dose and only take the next dose at your usual time. Do not take double or extra doses.